Wainua

/ Astra Zeneca

The recommended dose of eplontersen is 45 mg administered monthly. Supplementation at the recommended daily allowance of vitamin A is advised.
The pre-filled pen should be removed from refrigerated storage at least 30 minutes before use and allowed to reach room temperature prior to injection. Other warming methods should not be used.

Treatment of hereditary transthyretin-mediated amyloidosis (ATTRv
amyloidosis) in adults patients with Stage 1 and 2 polyneuropathy.

Hypersensitivity to the active substance or to any of the excipients

Vitamin A deficiency
Based on the mechanism of action, eplontersen is expected to reduce serum vitamin A (retinol) below normal levels. Serum vitamin A levels below the lower limit of normal should be corrected and any ocular symptoms or signs related to vitamin A deficiency should be evaluated prior to initiation of treatment.
During the first 60 days of pregnancy, both too high and too low vitamin A levels may be associated with an increased risk of foetal malformation. Therefore, pregnancy should be excluded before treatment initiation and women of childbearing potential should practise effective contraception. If a woman intends to become pregnant, eplontersen and vitamin A supplementation should be discontinued, and serum vitamin A levels should be monitored and have returned to normal before conception is attempted.
In the event of an unplanned pregnancy, this medical product should be discontinued. Due to the long half-life of eplontersen, a vitamin A deficit may even develop after cessation of treatment. No recommendation can be given whether to continue or discontinue vitamin A supplementation during the first trimester of an unplanned pregnancy. If vitamin A supplementation is continued, it should be given at the recommended daily allowance of vitamin A It is not known whether vitamin A supplementation in pregnancy will be sufficient to prevent vitamin A deficiency if the pregnant female continues to receive eplontersen. However, increasing vitamin A supplementation to above 3,000 IU per day during pregnancy is unlikely to correct serum retinol levels due to the mechanism of action of eplontersen and may be harmful to the mother and foetus.

Very common: Vitamin A decreased
Common: Vomiting, Injection site erythema/ pain/ pruritus, Proteinuria

No interaction studies have been performed. In vitro studies indicate that eplontersen is not a substrate or inhibitor of transporters, does not interact with highly plasma protein bound medicinal products, and is not an inhibitor or inducer of CYP enzymes.

Pregnancy: Eplontersen will reduce the plasma levels of vitamin A, which is crucial for normal foetal development. It is not known whether vitamin A supplementation will be sufficient to reduce the risk to the foetus. For this reason, pregnancy should be excluded before initiation therapy and women of child-bearing potential should practice effective contraception. If a woman intends to become pregnant, eplontersen and vitamin A supplementation should be discontinued, and serum vitamin A levels should be monitored and have returned to normal before conception is attempted. Serum vitamin A levels may remain reduced for more than 15 weeks after the last dose of treatment.
Lactation: It is unknown whether eplontersen or its metabolites are excreted in human milk. A risk to the breastfed child cannot be excluded. A decision must be made whether to discontinue breast-feeding or to discontinue/abstain from therapy, taking into account the benefit of breast feeding for the child and the benefit of therapy for the woman.

There is no specific treatment for an overdose with eplontersen. In the event of an overdose, supportive medical care should be provided.

If necessary, this medical product may be stored outside the refrigerator at a temperature below 30°C for up to 6 weeks in the original package.